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Diabetes Research Institute Foundation Spring 2021
“It’s a remarkable time for T1D research efforts worldwide as this investigational treatment enters the clinic.”
Clinical Trial for Novel Investigational Treatment of Type 1 Diabetes Open for Enrollment
Insulin-producing pancreatic cells derived from stem cells that could potentially provide an unlimited source of cells for islet transplantation.
Diabetes Research Institute investigators will lead the first site activated for the Vertex Pharmaceuticals patient study that will test VX-880, a novel investigational cell therapy with the potential to restore normal glucose control in those suffering from type 1 diabetes (T1D).
Committee Chair for the VX-880 clinical trial. “The field’s experience with the limited cadaveric islet transplants available, where some patients have experienced prolonged insulin independence for years, provides important proof-of-concept for the potential of cell therapy to be transformative for patients living with T1D.”
The program was granted Fast Track Designation by the U.S. Food and Drug Administration (FDA), which offers an expedited review process to accelerate treatments for urgent and unmet medical needs. It will evaluate the safety, tolerability and efficacy of VX-880 infusion in certain T1D patients. To be considered as a candidate for the trial, participants must have a clinical history of type 1 diabetes of at least five years, be 18-65 years of age, and have multiple episodes of severe hypoglycemia and impaired hypoglycemic awareness 12 months prior to enrollment.
The program’s groundbreaking work began in Dr. Doug Melton’s lab, who developed this line of insulin-producing pancreatic cells derived from stem cells. Vertex acquired Semma Therapeutics, the biotechnology company founded by Melton, in 2019.
“It’s a remarkable time for T1D research efforts worldwide as this investigational treatment enters the clinic,” said Camillo Ricordi, M.D., Director of the Diabetes Research Institute and Steering
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“I am grateful to the DRI team that will help with this unprecedented, first-in-human clinical trial,” added Ricordi. The University of Miami Health System was the first clinical site activated for this trial, that now also includes the University of Pennsylvania and Massachusetts General Hospital. Those interested in applying can contact the DRI’s clinical trials team at (305) 243-5321.